Findings

What do systematic reviews say?

7 questions · 7 findings · 2 reviews · Cystic Fibrosis and Genetic Disorders

No studies found 2 · 28.6%GRADE not reported 0 · 0%Very low 1 · 14.3%Low 4 · 57.1%Moderate 0 · 0%High 0 · 0%

Infusion reactions · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Infusion reactions · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Infusion reactions · adults with LOPD

It is very uncertain whether VAL‐1221 changes infusion reactions compared with alglucosidase alfa (very low certainty).

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
OR 2.8
1 study · 12 participants
Very low

Infusion reactions · 90 people with LOPD

Alglucosidase alfa showed no clear difference in infusion reactions compared with placebo (low certainty).

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
RR 1.21 [0.57, 2.61]
1 study · 90 participants
Low

Infusion reactions · adults with LOPD

Cipaglucosidase plus miglustat showed no clear difference in infusion reactions compared with alglucosidase alfa plus placebo (low certainty).

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
RR 0.94 [0.49, 1.8]
1 study · 123 participants
Low

Infusion reactions · adults with LOPD

Avalglucosidase alfa showed no clear difference in infusion reactions compared with alglucosidase alfa (low certainty).

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
RR 0.78 [0.42, 1.45]
1 study · 100 participants
Low

Infusion‐related reactions (number of participants with one or more reaction) · adults and children with mucopolysaccharidosis I

The intervention showed no clear difference in infusion‐related reactions (number of participants with one or more… (low certainty).

1 finding from 1 review